Cellular and gene therapy for major histocompatibility complex class II deficiency

被引:13
作者
Matheux, F [1 ]
Villard, J
机构
[1] Hop Cantonal Univ Geneva, Div Immunol & Allergol, Immunol & Transplant Unit, CH-1211 Geneva 4, Switzerland
[2] Univ Geneva, Sch Med, Dept Genet & Microbiol, CH-1211 Geneva, Switzerland
来源
NEWS IN PHYSIOLOGICAL SCIENCES | 2004年 / 19卷
关键词
D O I
10.1152/nips.01462.2003
中图分类号
Q4 [生理学];
学科分类号
071003 ;
摘要
Major histocompatibility complex (MHC) class II deficiency is a primary immunodeficiency. Lentiviral vectors are used for gene therapy in, a mouse model of this disease. In addition, by a direct genetic correction approach, a diagnostic test to determine which of the four MHC II genes is defective in new MHC II-deficiency patients has been optimized.
引用
收藏
页码:154 / 158
页数:5
相关论文
共 18 条
[1]   Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease [J].
Cavazzana-Calvo, M ;
Hacein-Bey, S ;
Basile, CD ;
Gross, F ;
Yvon, E ;
Nusbaum, P ;
Selz, F ;
Hue, C ;
Certain, S ;
Casanova, JL ;
Bousso, P ;
Le Deist, F ;
Fischer, A .
SCIENCE, 2000, 288 (5466) :669-672
[2]   Mice lacking the MHC class II transactivator (CIITA) show tissue-specific impairment of MHC class II expression [J].
Chang, CH ;
Guerder, S ;
Hong, SC ;
vanEwijk, W ;
Flavell, RA .
IMMUNITY, 1996, 4 (02) :167-178
[3]   Residual MHC class II expression on mature dendritic cells and activated B cells in RFX5-deficient mice [J].
Clausen, BE ;
Waldburger, JM ;
Schwenk, F ;
Barras, E ;
Mach, B ;
Rajewsky, K ;
Förster, I ;
Reith, W .
IMMUNITY, 1998, 8 (02) :143-155
[4]   RFXAP, a novel subunit of the RFX DNA binding complex is mutated in MHC class II deficiency [J].
Durand, B ;
Sperisen, P ;
Emery, P ;
Barras, E ;
Zufferey, M ;
Mach, B ;
Reith, W .
EMBO JOURNAL, 1997, 16 (05) :1045-1055
[5]   Primary immunodeficiency diseases: an experimental model for molecular medicine [J].
Fischer, A .
LANCET, 2001, 357 (9271) :1863-1869
[6]  
HACEIN BA, 2003, NEW ENGL J MED, V348, P255
[7]  
KLEIN C, 1995, BLOOD, V85, P580
[8]   A gene encoding a novel RFX-associated transactivator is mutated in the majority of MHC class II deficiency patients [J].
Masternak, K ;
Barras, E ;
Zufferey, M ;
Conrad, B ;
Corthals, G ;
Aebersold, R ;
Sanchez, JC ;
Hochstrasser, DF ;
Mach, B ;
Reith, W .
NATURE GENETICS, 1998, 20 (03) :273-277
[9]   Direct genetic correction as a new method for diagnosis and molecular characterization of MHC class II deficiency [J].
Matheux, F ;
Ikinciogullari, A ;
Zapata, DA ;
Barras, E ;
Zufferey, M ;
Dogu, F ;
Regueiro, JR ;
Reith, W ;
Villard, J .
MOLECULAR THERAPY, 2002, 6 (06) :824-829
[10]   Expression of the three human major histocompatibility complex class II isotypes exhibits a differential dependence on the transcription factor RFXAP [J].
Peretti, M ;
Villard, J ;
Barras, E ;
Zufferey, M ;
Reith, W .
MOLECULAR AND CELLULAR BIOLOGY, 2001, 21 (17) :5699-5709